We have a diversified and balanced clinical development pipeline that covers multiple therapeutic areas, including CNS, cardiovascular and immunological disorders, as well as orphan diseases.

Compound Mechanism of Action Target Indication Status More information
Daridorexant Dual orexin receptor antagonist Insomnia Filing in preparation

Investor webcast (first study)

Investor webcast (second study)

Aprocitentan*  Dual endothelin receptor antagonist Resistant hypertension management Phase 3 Investor webcast
Clazosentan Endothelin receptor antagonist Vasospasm associated with aneurysmal subarachnoid hemorrhage (aSAH) Phase 3 Investor webcast
Lucerastat Glucosylceramide synthase inhibitor Fabry disease Phase 3 Investor webcast
Selatogrel P2Y12 receptor antagonist Suspected acute myocardial infarction Phase 3 in preparation  
Cenerimod S1P1 receptor modulator Systemic lupus erythematosus Phase 2  
ACT-774312 CRTH2 receptor antagonist Nasal polyposis Phase 2  
ACT-539313 Selective orexin 1 receptor antagonist Psychiatric disorders Phase 2 in preparation  
GBA2/GCS inhibitor Rare lysosomal storage disorders Phase 1 complete  
ACT-1004-1239 - Immunology / Cancer Immunotherapy Phase 1  
ACT-1014-6470 - Immunology Phase 1  
ACT-541478 - CNS Phase 1  

* In collaboration with Janssen Biotech to jointly develop aprocitentan, Janssen Biotech has sole commercialization rights worldwide

Neurocrine Biosciences has a global license to develop and commercialize our ACT-709478, a novel T-type calcium channel blocker, for the treatment of a rare form of pediatric epilepsy. In May 2020, Neurocrine announced plans to initiate a Phase 2 study for ACT-709478 in the second half of 2020.